European Scleroderma Observational Study

Date: Tue 25th April 2017

Treatment outcome in early diffuse cutaneous systemic sclerosis

This was a project co-funded by Scleroderma & Raynaud's UK (SRUK) and EULAR. It was conducted by Xiaoyan Pan and colleagues at Hope Hospital, Salford (published in Scleroderma News, November 2014)

In the 'diffuse' form of scleroderma, skin thickening can progress fairly rapidly, usually starting in the hands, face or feet but then spreading upwards to involve upper arms, thighs, or trunk. Those affected are also at high risk of developing lung, kidney or heart involvement especially in the first three years of the illness. A number of different drugs with effects on the immune system (called 'immunosuppressants) are currently used by doctors in the treatment of early diffuse scleroderma. However, all can have side effects and the efficacy of the different drugs is not fully established.

The objective of the European Scleroderma Observational Study (ESOS) is, by careful recording and analysis, to examine the effectiveness of different immunosuppressant drugs currently favoured by doctors in treating scleroderma. Information obtained through ESOS will, in the future, help to inform doctors' treatment decisions.

ESOS is supported as part of the EULAR (European League Against Rheumatism) Orphan Disease Programme which aims to further knowledge of why scleroderma develops, and how best to treat scleroderma. To allow enough patients to be recruited into the study to provide meaningful results, ESOS opened 44 recruiting centres in 16 European countries between 2010 and 2012.

The approval processes for the different countries took longer than planned, with the result that recruitment was initially under target. The study protocol was therefore amended and opened another six recruiting centres in Austria, Canada and USA. ESOS was also publicised at international conferences to facilitate the recruitment. Despite this, a 12-month extension of recruitment was essential to hit target recruitment.

Funding from SRUK allowed the ESOS recruitment period to be extended by one year. Target recruitment was then completed, with 322 patients recruited.

Who led the research: The international steering committee; the study was coordinated by Xiaoyan Pan at the Centre for Musculoskeletal Research within the University of Manchester

Our Funding: £35,000

Duration: 12-month extension (full study 2010-2016)

Official title of the application: European Scleroderma Observational Study

Tags: scleroderma, European Scleroderma Observational Study, international